There is a boy who stole my heart at first site and I will not let Duchenne Muscular Dystrophy steal him!

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Ethan wrote a wonderful piece on what it’s like for us to raise a child with a terminal illness. I have added below. I know a lot of people don’t like me saying terminal illness but that is what it is. He isn’t “sick”. It won’t “go away.” I don’t want to trivialize it. Unless something changes-it’s terminal. We live with that notion every day. We choose to live life. I am lucky I have friends and family who truly care and have decided to take the journey with us, no matter how scary, difficult or painful. When I say we are lucky, I mean it. To steal from another DMD family I admire – #makeeverydaycount!

Here is Ethan’s beautifully written explanation.

People often ask us (Peyton’s Parents) how is Peyton doing. How do you answer that? By no means am I upset with the question so often asked. It’s an obvious question that I would ask myself. I usually answer it by saying, “He seems the same.” However, how do I really answer that question. I’ll try an explain it with this. We don’t really know what is happening inside his body, nor do the doctors. There is no cure to this disease. Heck, there really isn’t any treatment. We are working with “maybes,” “hopefullys,” “don’t know if this works but we will try it.” The conclusion is always the same with DMD. Some boys have very short lives, 10 years and even younger. Others live into their 30s and 40s. But the average boy is in a power wheelchair around the age of 12 and has an average life of 24-25. Just writing this and thinking about it is difficult.

The average DMD boy enters mid-life about Peyton’s age. See, Peyton is in the phase of the disease (DMD) known as the “Honeymoon” period. He is still walking, appears to be doing well and seems ok to the average person. This is very common with DMD. Just like with cancer, when people can see you are not doing well, it is too late. As parents, it is extremely difficult to cope knowing that your child’s body is deteriorating every minute and there is nothing you can do to stop it. As a parent, this disease is on your mind consistently, as if raising kids didn’t give enough to worry about.

I posted this article because I think it is great and it explains why our kids are always off on some adventure. Parents have a few ways to deal with DMD. Denial, bathe in self-pity and sorrow or get all the life experiences in that you can in a short period of time. DMD forces you to re-evaluate what is important in life. It assists you by changing your perspective and how you view the world. Staying late at work and putting in extra hours to “Get Ahead” may not seem as important as it once did. “Getting Ahead” now means spending as much time with your children as you can. You understand that life is going to get tougher as the disease progresses. You also understand that you only have one chance to raise your children, they won’t be young for long.

If you know us or have seen some of our recent posts, you obviously know we have chosen to live-life and experience as much as we can TODAY. That’s right, TODAY. This is something you should also consider even if your child is healthy. Everyday across America and the world, parents often lose children to variety of tragic situations. It should remind all of us that we have been given no guarantee to a long life.

Today is World Duchenne Awareness Day. I wish I had no idea what Duchenne was. Unfortunately our lives turned upside down one January day a couple of years ago. Our middle son Peyton was diagnosed with Duchenne Muscular Dystrophy (DMD) at the age of 6. He is now 10 years old. DMD is an aggressive, degenerative, and terminal childhood muscle wasting disease affecting boys. It is extremely rare (but possible) for girls to have DMD.

When most middle school children are preparing for teenage years and adulthood, the natural history of this disease is:

loss of ambulation and being bound to a power wheelchair at the age of 12

full loss of mobility (including ability to feed oneself) in the mid to late teens

tragically short life expectancy by age 24

This is a horrible, debilitating disease that no child should have to endure. Ethan and I would gladly trade places with him if it were possible. DMD is often a genetic disease unknowingly carried by the mother. There are cases, such as ours, where the mother is not a carrier and the disease is caused by a random genetic mutation. Unfortunately, there is no cure. Medically researchers are in the infancy of the development for a treatment.

Despite all of this, there is hope. January 2015, Peyton was admitted into a clinical trial for a drug called Eteplirsen. This drug is meant to slow the progression of the disease and it is the first of its kind. It is by no means a cure or a perfect treatment. Part of Peyton’s DNA and genetic code for muscle-building is missing. DMD patient’s bodies do not produce a protein call dystrophin. In the DMD world, everyone knows which code their child is missing. Peyton is missing exons 45-50. It is actually very complicated and Peyton happens to be in the biggest block, known as EXON-51. EXON 51 happens to be approximately 13% of all the DMD patients.

Based on Eteplirsen, we now have hope that our child will live longer and have a better life. He is one of the lucky ones. This drug is very expensive, we have been told Eteplirsen ranges in price between $2000 – $5000 for each treatment, each week, for each child, indefinitely. Peyton goes to UCLA weekly to have the drug infused through a port in his chest. We have allowed him to make the choice and Peyton chose to be a human guinea pig in hopes that this will not only help him, but thousands, if not millions, of other DMD children. We believe this new approach to combating DMD will ultimately help others with different diseases.

There are twelve boys who have been on the drug for over 4 years and 10 are still walking into their teens. This is unheard of in the DMD community. We have seen positive results in Peyton. Not only has the disease progression stabilized, but he is actually getting stronger and there have been no side effects whatsoever.

In April of this year, researchers, doctors, advocates and DMD families, including Ethan and Peyton, descended on Washington D.C. to plead our case for the approval of Eteplirsen to the FDA. We are still waiting patiently, over 100 days past the deadline set by the FDA, for an answer. A denial of this drug will be devastating for our community and a death sentence for Peyton. We wish we were being dramatic, but that is the absolute truth and something that haunts us every day. We have hope that they will do the right thing and approve Eteplirsen. If approved, the pipeline opens up for other kids to have access to the drug.

The United States Congress and United States Food and Drug Administration (FDA) have created guidelines for “Compassionate Use” and the Orphan Drug Act (ODA). Duchenne Muscular Dystrophy is an orphan disease because it affects such a small population (under 200,000) compared to other diseases such as cancer. A normal drug takes on average twelve years to get through the FDA process for safety and effectiveness. Under those guidelines, that is a death sentence to DMD boys. The drugs we have today will not reverse the consequences of muscle wasting due to waiting for the FDA and years of data. It took approximately 25 years of data to show that steroids had a positive effect on DMD patients. In 2005, steroids became standard care for DMD patients.

We can only speculate as to the reason the drug has not yet been approved since it is working. From our perspective, there appears to be a bureaucratic battle within the FDA. The FDA is over 90 days past the deadline they set for a decision. One of the big reasons some in the FDA are against approval for Eteplirsen is to protect against the approval of a drug that does not work, known as a “Type I error.” However, what about failing to approve a drug that does work. That is known as a “Type II error.” In a recent article by Alex Tabarrok, “Is the FDA Too Conservative or Too Aggressive?” Tabarrok stated, I have long argued that the FDA has an incentive to delay the introduction of new drugs because approving a bad drug (Type I error) has more severe consequences for the FDA than does failing to approve a good drug (Type II error). In the former case at least some victims are identifiable and the New York Times writes stories about them and how they died because the FDA failed. In the latter case, when the FDA fails to approve a good drug, people die but the bodies are buried in an invisible graveyard.

Several people we have met during this process have provided many theories as to why the drug has not been approved, those mostly being politics and big money. FDA may want to protect the bureaucratic process. The drug is expensive, how would that effect health insurance and will it pave the way for more expensive drugs for other orphan type diseases. The company that has developed Eteplirsen is a small drug developer. A denial of the drug could devastate the bottom line for the company and therefore make it cheap to purchase. It takes approximately one billion dollars to bring a drug to market. If a small company has the ability to enter the market, it creates more competition for large drug companies that are not as efficient. It also decreases the barriers to entry for drugs in America. It bypasses many of the protocols normal drugs have to pass because Eteplirsen treats a terminal orphan disease without another treatment. Many medical school administrators may not be as willing to advocate for Eteplirsen due to grants and research funding provided by large drug companies. In actuality, we really do not know the reasons behind the delay, all we know is that boys are still dying.

Soon after Peyton’s diagnosis we founded Peyton’s Project. We were devastated by the diagnosis but decided we were going put our efforts in to making a difference and his life mattered. Our goal is to raise money to fund research for DMD as well as provide quality of life equipment to kids who don’t have the means or access. This is a VERY expensive disease. Their life span may be short but it doesn’t have to be isolating and lonely when there are devices that allow them to participate in life instead of watching from the sidelines. Unfortunately, most families don’t have the means to provide these devices and insurance won’t pay for them.

When it comes down to it, we are parents. A Mom and Dad trying to save our son’s life; trying to raise Peyton’s brothers with compassion and understanding without getting lost in the shuffle; trying to work two demanding jobs so we can provide a better quality of life for our boys; trying to make a difference in the life of our child and the lives for all the other families and children affected by this aggressive disease. Raising money for research and adaptive equipment is just a small way we can give back to the DMD community that embraced us when we were reeling from this diagnosis and continues to embrace us in our journey.

Peyton is a beautiful child with a heart of gold and a smile that lights up a room and leaves an impression on your heart. Once you meet him, you will never forget him. He is worth the fight and we thank you so much for your support!

Over the summer Peyton and I have been spending a lot of time together in the car. Anyone who knows Peyton, knows he’s wise beyond his years. Sometimes I have to remind myself he is only 10. The car seems to be the place he does most of his talking.

A couple weeks ago out of no where he asks me why anyone would want to wear a condom. I pretty much almost drove the car off the road and wondered why they always ask me these questions? I had boys-shouldn’t they be asking their father? Nope-always me, always when I am driving, always when I am the least prepared. I muttled myself thru an answer of diseases, pregnancy, monogamy, abstinence, love, respect, blah, blah, blah – all the while trying not to throw up.

The next day around the very same stretch of freeway, he asked me why he had Muscular Dystrophy? Why his DNA was bad? Why he couldn’t run like his friends? Why he can’t be a normal boy? Why won’t he grow taller? Why he can’t eat what he wants? Why is he gaining weight? Why is his face so round? – It was a barrage of questions. Each one another kick in my gut.

Answers were fleeting. These are tough conversations no parent should have to have with their child. No child should have to think about. As he gets older and more aware of his disease I know there will be more questions, more difficult conversations I am woefully unprepared for.

That night as I was telling Ethan about it I told him – I think I would much rather discuss condoms.

I spent the last 2 weeks in a dorm with strangers to train with Peyton and his new canine companion. The families were all awesome and I am glad our paths crossed. They elected me class speaker. These were my words.

My name is Bonnie Marquez and I am representing the graduating class of February 2016. We came together 2 weeks ago with our own preconceived ideas of what we needed or wanted but we all had a similar goal-to find the perfect companion for our self, our child or our workplace. I can honestly say … mission completed. Team training has been an amazing time of bonding with new friends, trainers and our dogs. After a couple of days of meeting and working with several potential dogs, pre-match day came. It was a thrill to watch each and every team find their FUREVER companion. Each one couldn’t have been more perfectly matched.

Chance will be the most popular kid in town with Neelia! She gives him confidence and a purpose that will carry over into other areas like school, therapies and daily life. Chance and his family are so appreciative to have Neelia in their life and can’t wait to get to know her better.

Mitchell’s mother was delighted when he actually reached his hand out for Rockford. Rockford provides Mitchell with improved self-confidence; as it changes how others view him and how he views himself. Rockford provides emotional support at medical appointments and pride that comes from the responsibility of caring for him. Rockford helps encourage Mitchell’s speech by voicing commands and providing a bridge for conversation in social situations. Not to mention how helpful Rockford will be in retrieving frequently dropped items. Mitchell’s disability isolates him, so for Rockford to offer mutual unconditional love and companionship means the world to his family.

Barbara fell in love instantly with Poehler! His sweet, playful personality engages Barb and his tender side fulfills her need for companionship. On pre-match day he greeted Barbara by plucking her bow off her head and sitting on her lap… he chose his girl! He marches proudly alongside Barb’s wheelchair, snuggles up to her with all of her equipment on and is retrieving his toys and dropping them in her lap. He is hard-working, brilliant, silly and sweet. He will be Barbara’s companion, friend, arms and legs and her pride and joy! Poehler is our dream come true!

Sabine’s companion Balter not only provides companionship but helps her with everyday tasks.

Adam’s family feels very fortunate to have Blade come in to their life. He is such a loveable and responsive dog. He is always there for Adam. Often Blade sits next to Adam and rests his paw on him. This is very helpful especially when Adam has to do his oral exercises, which are not his favorite. Adam loves to talk to Blade every night before going to sleep and share with him his day. Blade has been such an added blessing to their family.

Kingston is Devin’s 2nd Canine Companion. Ellie was going to be hard to replace but Kingston and Devin made a connection the very first meeting. Kingston was the only dog to get right on Devin, look him in the eye and not care about anyone or anything going on around him. Devin knew instantly this was his dog to the extent his parents were worried if he didn’t get him how devastated he would be. Kingston is Devin’s Best friend. He’s also his chick magnet. Devin and Kingston are just 2 guys hanging out watching the Broncos, Dodgers and living it up in Vegas!!!!! Kingston is gentle giant with two gears: loving and sensitive and full-fledged fetch player. We all chuckled when Michelle asked if these dogs were fully grown and Patrick said-Thank God!!!!

Barb (the dog-not the child) will begin her new life beside Susan and Fawn working within the Courthouse. When Barb is working she is very serious and stoic. Susan and Fawn were so pleased one morning when they greeted her at her kennel and she was so excited to see them her whole crate was shaking. It really showed them both sides of her personality and has convinced them that she will be perfectly placed in the courthouse facility. She can be serious in court and still have fun with the kids and make them enjoy the process of meetings and court.

Hani will take her place beside Beth in the classroom. She will help students with disabilities by providing them the opportunity to care for and be responsible for something other than themselves. Hani’s social personality makes her perfect for this environment.

My son, Peyton, was matched with Beatrice. “Princess Bea” as I call her. The first time I walked with her I told her “let’s go” she looked me in the eye, turned and pranced beside me and I knew this was our girl. There was an instant connection. Peyton has Duchenne Muscular Dystrophy which is an aggressive muscle wasting disease. He spends a minimum of 1 day per week in the hospital at UCLA, sometimes more. That can be lonely and isolating as he misses a lot of school and social activities. Bea is a game changer for our family. She provides motivation for him to move around while playing fetch. She provides and ice breaker when people are unsure of his disability. Most of all, she provides a constant, unconditional loving and accepting BFF for Peyton. Watching her snuggle on the bed with him each night melts my heart.

We couldn’t be more grateful for Canine Companions for the gift they have given all of us.

The last 2 weeks have been challenging, intense and most of all, rewarding. All of us put our lives on hold, leaving spouses, children and jobs to live in a dorm with strangers. Turns out, this group couldn’t have been better. Everyone has been incredibly helpful, supportive and encouraging. I am going to miss the morning and evening camaraderie between all the families.

So thank you to everyone who had a hand in guiding our fur babies to this point…the puppy breeders and the puppy raisers whose devotion to these dogs and program is commendable. To Simi and everyone at Canine Companions who make this program successful. You have made us feel incredibly welcome.

I cannot end without thanking Kyle and Grace for being wonderful trainers and teachers. Their love of these dogs and their craft is apparent. The look of love Bea has when she sees Grace and Poehler has for Kyle is a testament to their commitment. They have been patient, helpful, stern when necessary, reassuring and the reason we have been successful.

Today marks the first day in a lifetime of love and companionship between our dogs and their companions. We thank you all for sharing in our day. Let’s celebrate!

I realize I haven’t updated in awhile. I have so many emotions running thru me. When I get a chance I will catch you all up on this roller coaster of a year. We have strapped ourselves in as I don’t see us getting off any time soon.

Earlier this year (although it feels like longer) I asked you to help us by signing a petition to push the FDA to approve a drug that has the very real potential to help Peytie. I asked and you delivered. You signed, you told your family and friends to sign and thanks to you, MY PRECIOUS LITTLE BOY HAS A CHANCE! Although the drug wasn’t approved, it was sent back to clinical trial and Peytie started the recruitment process this week.

So thank you for caring, thank you for sharing and thank you for loving my family. As you end the year know that you did your part to help not only my family and son, but many, many more.

Come help us raise a lot of $ for the boys with the awful disease called Duchenne Muscular Dystrophy (DMD). We will have a ton of adorable animals to play with, pumpkins for sale, face painting, giant board games and so much more. The raffle items are amazing: Disneyland, Legoland, Annual passes to Magic Mountain, Dinner Party with a personal chef, tickets to We will Survive at the Hollywood bowl with Taylor Swift, Pharrell Williams, Ariana Grande, Lady Antebellum, Iggy Azalea, Paramore, Sia and special guest Gwen Stefani and SO MUCH MORE!!!! See you October 11th. Info below: